re: Human Gene Therapy

The other part that makes it harder is the timeline of the gene insertion. i.e. will simply replacing a protein correct/improve the problem or has someone already missed out on the appropriate developmental steps as a result of it since so many things on the cellular level are orchestrated by the local milieu (not speaking to your case in specific as I'm really not familiar with retinoschisis, I'm not an optho).

Really excited to see where gene replacement therapy takes us in the future but anticipate it will be like most other things where we have plenty of unintended consequences that we learn even more from. It's how science works.